FDA de­rails Acor­da's mar­ket­ing pitch, cre­at­ing a cri­sis as flag­ship drug founders

Acor­da just en­tered a world of hurt.

With its patent pro­tec­tion crum­bling around its main­stay drug, the FDA has re­fused to file its ap­pli­ca­tion for CVT-301, the res­cue pro­gram that was des­ig­nat­ed to take the lead drug’s place. The RTF cre­ates an em­bar­rass­ing and per­haps crip­pling prob­lem for the biotech, which is fight­ing a last-ditch bat­tle to main­tain patent pro­tec­tion on its one big drug, Ampyra.

Ron Co­hen

Ac­cord­ing to Acor­da, the FDA says the com­pa­ny’s mar­ket­ing ap­pli­ca­tion, rushed to reg­u­la­tors as the com­pa­ny re­struc­tured and down­sized, is in­com­plete. Reg­u­la­tors said they were un­able to do a prop­er re­view as they don’t know what date Acor­da’s man­u­fac­tur­ing cen­ter will be ready for in­spec­tion and have a “ques­tion re­gard­ing the sub­mis­sion of the drug mas­ter pro­duc­tion record.”

Acor­da re­port­ed that the FDA al­so re­quest­ed ad­di­tion­al in­for­ma­tion at re­sub­mis­sion, which was not part of the ba­sis for the RTF.

Its shares quick­ly plunged 26% on the news.

Nor­mal­ly, a de­lay like this would be seen as a sticky prob­lem, but one that could po­ten­tial­ly be re­solved with­out huge de­lays. The prob­lem for Acor­da, though, is that the com­pa­ny has vir­tu­al­ly no lee­way for a sna­fu like this. Not on­ly does it leave Acor­da’s ex­ec­u­tive team with a black eye on ex­e­cu­tion, it cre­ates se­ri­ous doubts about its abil­i­ty to quick­ly and ef­fi­cient­ly line up a re­place­ment drug for its sales team by next sum­mer, as the lit­i­ga­tion over its patents for Ampyra comes to a head.

Leerink’s Paul Mat­teis found the whole thing puz­zling and dis­con­cert­ing, con­sid­er­ing the like­li­hood of a loom­ing ap­proval. And he still has ques­tions about what is go­ing on at Acor­da.

It seems like the best case sce­nario would be a re-sub­mis­sion around the end of this year, and its en­cour­ag­ing that FDA did not make any re­quests re­gard­ing the ef­fi­ca­cy or safe­ty da­ta. Nonethe­less, even af­ter speak­ing with the com­pa­ny, the RTF is mys­te­ri­ous to us, and we ex­pect the stock to be un­der pres­sure un­til opac­i­ty is lift­ed.

As I re­port­ed in June, while re­view­ing the late-stage da­ta sub­mit­ted to the FDA on the in­haled lev­odopa, any set­back on CVT-301 would be a cat­a­stro­phe for Acor­da.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

Now Acor­da is left to des­per­ate­ly seek a quick meet­ing with reg­u­la­tors to clear up any of these ques­tions. But the FDA is not known for scram­bling a re­sponse in any of these sit­u­a­tions, leav­ing Acor­da’s time­line in the air just when it most needs cer­tain­ty. Just weeks ago one of its biggest in­vestors pushed CEO Ron Co­hen to auc­tion the com­pa­ny off rather than risk the con­se­quences of fail­ing at its high wire act. The com­pa­ny pushed back, but a sale may come back on the ta­ble now whether or not it likes the idea.

“We will work with the FDA as quick­ly as pos­si­ble to ad­dress the open is­sues and to clar­i­fy the path to suc­cess­ful­ly re-file our ap­pli­ca­tion,” said Co­hen in a state­ment. “We re­main con­fi­dent in IN­BRI­JA’s da­ta pack­age and its promise as an im­por­tant new ther­a­py for peo­ple with Parkin­son’s dis­ease. We see tremen­dous long-term val­ue in its sol­id clin­i­cal pro­file, sig­nif­i­cant com­mer­cial op­por­tu­ni­ty and strong IP, and we re­main fo­cused on work­ing to bring pa­tients this im­por­tant new ther­a­py.”

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.

BeiGene's new website helps direct cancer patients and caregivers to a wide variety of sources for help.

BeiGene re­veals men­tal health and can­cer care gap in study, de­buts dig­i­tal re­sources

One-fourth of cancer patients are living with depression — and another 20% suffer from anxiety. That’s according to new study results from BeiGene, conducted by Cancer Support Community (CSC), about the mental and emotional health of cancer patients.

While the fact that people with cancer are also dealing with depression or anxiety may not be surprising, what is — and was to BeiGene — is that a majority of them aren’t getting support. 60% of respondents said they were not referred to a mental health professional, and even more concerning, two in five who specifically asked for mental health help did not get it. CSC, a nonprofit mental health in cancer advocacy group, surveyed more than 600 US cancer patients.

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One of the paintings from Gilead's latest campaign making AI art to help MBC patients be 'seen and heard.'

Gilead com­bines ar­ti­fi­cial in­tel­li­gence and art to draw at­ten­tion and hope to MBC

What if you could “see” the emotions and feelings of people living with metastatic breast cancer? That’s what Gilead Sciences’ agency VMLY&R Health did last year, using artificial intelligence and sound analytics to turn the interviews of three women living with metastatic triple-negative breast cancer into works of art.

Using the sound waves, a robotic painting device translated their stories of struggle and hope into colors, contours and brush strokes. The result? An art exhibition called “Paintings of Hope” that was first displayed at ESMO in September in Paris, but has since traveled to hospitals and medical conferences in Europe and Spain.

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